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Vertex Pharmaceuticals has been in business for more than 20 years, and burned through more than $2.8 billion on a quest to develop drugs that shake up the medical standards of care. Now in the coming weeks and months, it will get the first definitive evidence that will say whether its lead drug candidate for hepatitis C has achieved its goal.

Investors, looking at data from preliminary and mid-stage clinical trials, have already pumped up the market value of Vertex (NASDAQ: VRTX) past $7.5 billion largely in anticipation that its drug, telaprevir, will transform the lives of patients with the chronic liver-damaging condition. But the operative word there is preliminary. Vertex, which is based in Cambridge, MA, and has significant operations in San Diego, is still waiting for its first proof from Phase III clinical trials, the final step of testing required by the FDA before a drug can go on sale in the U.S.

These pivotal trials began two years ago, and enrolled more than 2,000 patients combined in three studies. The results are completely blinded to doctors, patients, investors, and people at Vertex. To say all parties are in suspense for these results would be an understatement. They can’t wait to get their hands on the new data and start digging through it.

Vertex has generated the excitement around what could be a first-in-class protease inhibitor drug for hepatitis C. It has excited researchers because it has been able to double the cure rate while enabling patients to cut their standard course of treatment time in half. That means that many more of the 3 million Americans and 170 million people worldwide with chronic hepatitis C liver infections will be likely to seek out treatment, and be able to stand up to the side effects of standard therapy that causes flu-like symptoms. If Vertex can prove this idea once and for all in the three pivotal trials, Vertex could seek FDA approval later this year and bring telaprevir to the market in 2011. U.S. sales alone could amount to more than $2 billion after a couple years, analysts say.

Bob Kauffman

To help our readers get ready for this data, I spoke to Kauffman for a refresher on what the three big Vertex trials were designed to ask and answer. The key point to watch for in all of these studies is what is called a “sustained viral response,” or SVR, which is recorded when researchers can’t find any sign of virus in a patient’s blood sample for a full 24 weeks after they completed their course of therapy. This is the gold standard measurement for all hepatitis C drugs, and is commonly known as a “clinical cure.”

The first study to watch for is called “Advance.” This trial, started in March 2008, enrolled 1,050 patients who had never been treated before for hepatitis C—a so-called “naïve” patient population. This study is essentially designed to confirm earlier trials called Prove 1 and Prove 2, Kauffman says. Patients either got the Vertex drug in combination with standard treatments for 24 weeks, or the standard treatments for the usual 48 weeks. The trial is designed to ask whether patients can stop treatment early with the Vertex drug, so they can avoid having to put up with the flu-like side effects of pegylated interferon alpha and ribavirin, Kauffman says. Results from that study should be available by the end of June, he says.

Not long after that data arrives, Vertex plans to hear results from two other key trials before the end of September. One of them, called “Illuminate,” is enrolling patients who have never before … Next Page »

Telaprevir will be used in a “triple therapy” along with pegylated interferon and ribavirin. The problem is interferon. It is a very dangerous drug and has caused permanent nerve damage, memory problems and neuropsychiatric disorders to many people, including me. I wish the FDA would do an investigation on just how many people, were damaged by interferon. Do a google search. Read the Hepatitis C forums. You will see alot of people that was ok, before they started the treatment, and afterward was left with numerous debilitating medical problems. I wish I could sue the makers of this horrible drug interferon. It’s been two years since treatment and I live everyday in pain. Frustrated in Texas…..!

I am literally dying with hepatic encephalopathy and have developed cirrhosis with ammonia levels that will not come down. I failed the interferon combo and the side effects were horrible, but I cannot wait to try this drug out with all the side effects. Transplant does not work as you become infected again. I am counting the minutes to try and trying to hold on. I want to go back to work, I want to not be sick, I want to grow old (I’m just 51). I would do anything to have my health back.

I did a year on Peg-Intron (alph interferon 2-b). I’ve been in pain ever since. My legs have an excruciating vibration that never quits. It varies in intensity. At best it’s a very uncomfortable tremble in my lower legs. But most of the time it is much worse. Often consuming most of my entire body. Sometimes from my ribcage to my toes. My legs are also in constant pain. The vibration causes the muscles, tendons, and joints in my legs to hurt costantly, because they are always tense and trying to compensate. I’m just 58, I would also do anything to have my health back. I want my LIFE back. I have to go lay down now. Too much activity causes more pain. I wanna get out of bed, go to work. I want to go sailing again. But I doubt I ever will. Interferon screwed me up so much that IF I ever try another drug treatment and it doesn’t help me I hope it kills me. Sounds very dramatic, but that’s exactly how I feel. Depression was a battle during treatment. It’s worse now! I think about suicide. But it’s not an option, I have a family.